The FDA granted breakthrough therapy designation to ruxolitinib for the treatment of patients with acute graft-versus-host disease (aGVHD), a condition that has no approved treatments specific for the disease. The decision was based on promising results from a retrospective analysis of patients who developed corticosteroid-refractory aGVHD following allogeneic hematopoietic cell transplantation for a hematologic malignancy.

The FDA granted breakthrough therapy designation to ibrutinib monotherapy for the treatment of patients with chronic GVHD (cGVHD) who have not responded to one or more lines of systemic therapy. Ibrutinib also received Orphan Drug Designation for cGVHD. These decisions were based on results from a phase Ib/II study, where ibrutinib demonstrated early clinical activity in the reduction of cGVHD based on the National Institutes of Health Consensus Response Criteria.